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NHS gives the green-light to a “gene-silencing” drug for the first time


Key points from article :

NHS gives approval for the first time for a “gene-silencing” medicine to be used.

Can be used to reverse amyloidosis, a fatal disease that can cause nerve and organ damage.

Messenger RNA copies the code from DNA and provides instructions to make new proteins.

The new drug patisiran causes RNA interference and kills the messenger RNA.

Effectively silences the rogue transthyretin gene and lowers levels of amyloid protein in the body.

Potential to treat diseases such as porphyria, Alzheimer’s and Parkinson’s using gene-silencing.

Study published in the New England Journal of Medicine.

It took thirteen years for RNA interference technology to come to this stage

Mentioned in this article:

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Julian Gillmore

Reader in medicine/honorary consultant nephrologist at Royal Free Hospital.

The New England Journal of Medicine

Scientific Journal devoted to medical research.